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beta thalassaemia

This page shows the latest beta thalassaemia news and features for those working in and with pharma, biotech and healthcare.

AstraZeneca forms $2bn gene silencing pact with Silence Tx

It is also working on its own in-house projects, namely SLN124 for beta thalassaemia, which is in phase 1b testing, and SLN360 which targets Lp(a) and is in early

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Incyte launches award programme recognising innovation in GVHD community

Other developers are aiming to produce therapies which avoid the causes of GVHD – that includes bluebird bio, whose beta thalassaemia treatment Zynteglo/LentiGlobin is designed to avoid the complications of GVHD
Bluebird bio leans on pipeline and Zynteglo launch as revenues fall

Aside from the mixed figures, bluebird is looking forward and focusing on the launch of its recently approved beta-thalassaemia gene therapy Zynteglo (autologous CD34+ cells encoding βA-T87Q-globin gene). ... The one-off gene therapy was approved by the
CRISPR gene-editing in cancer patients is safe, according to new study

Both have severe haemoglobinopathies – including transfusion-dependant beta thalassaemia (TDT) and severe sickle cell disease (SCD).
Bluebird bio launches beta thalassaemia gene therapy Zynteglo in Germany

The one-off gene therapy was approved by the European Medicines Agency (EMA) last June, for patients 12 years and older with transfusion-dependent beta thalassaemia (TDT) who have no matching ... Zynteglo (autologous CD34+ cells encoding β
Bluebird bio’s sickle cell gene therapy shows early-study benefit

Closely follows approval of beta thalassaemia therapy Zynteglo. Bluebird bio’s gene therapy for sickle cell disease (SCD), LentiGlobin, has demonstrated significant benefit in a small group of patients treated in ... The two patients both had severe

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Case-study for thalassaemia: Dimitri Azzopardi

An in-depth look at beta thalassaemia for Rare Disease Day. Beta thalassaemia major is a serious, lifelong genetic blood disorder. ... At least 75, 000 children are born. with thalassaemia worldwide every year, of which 10, 000 have the debilitating form
20 for 2020 – Five pharma companies to watch

treatment ozanimod and myelodysplastic syndromes/beta thalassaemia candidate luspatercept – to deliver on their potential.

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